Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2018 | Apr. 30, 2018 | |
Document and Entity Information [Abstract] | ||
Entity Registrant Name | AKCEA THERAPEUTICS, INC. | |
Entity Central Index Key | 1,662,524 | |
Document Type | 10-Q | |
Document Period End Date | Mar. 31, 2018 | |
Amendment Flag | false | |
Current Fiscal Year End Date | --12-31 | |
Entity Well-known Seasoned Issuer | No | |
Entity Voluntary Filers | No | |
Entity Current Reporting Status | Yes | |
Entity Filer Category | Non-accelerated Filer | |
Entity Common Stock, Shares Outstanding | 85,593,246 | |
Document Fiscal Year Focus | 2,018 | |
Document Fiscal Period Focus | Q1 |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) - USD ($) $ in Thousands | Mar. 31, 2018 | Dec. 31, 2017 |
Current assets: | ||
Cash and cash equivalents | $ 69,907 | $ 58,367 |
Short-term investments | 175,028 | 201,763 |
Contracts receivable | 0 | 5,413 |
Other current assets | 5,628 | 1,302 |
Total current assets | 250,563 | 266,845 |
Property, plant and equipment, net | 50 | 77 |
Licenses, net | 1,191 | 1,221 |
Deposits and other assets | 661 | 661 |
Total assets | 252,465 | 268,804 |
Current liabilities: | ||
Accounts payable | 2,578 | 2,381 |
Payable to Ionis Pharmaceuticals, Inc. | 27,737 | 14,365 |
Accrued compensation | 3,773 | 4,083 |
Accrued liabilities | 13,413 | 7,570 |
Current portion of deferred revenue | 48,866 | 58,192 |
Other current liabilities | 1,929 | 1,875 |
Total current liabilities | 98,296 | 88,466 |
Long-term portion of deferred rent | 10 | 12 |
Long-term portion of deferred revenue | 7,859 | 12,501 |
Total liabilities | 106,165 | 100,979 |
Stockholders' equity: | ||
Common stock, $0.001 par value; 100,000,000 shares authorized at March 31, 2018 and December 31, 2017; 66,803,803 and 66,541,629 shares issued and outstanding at March 31, 2018 and December 31, 2017, respectively | 67 | 67 |
Additional paid-in capital | 472,549 | 464,430 |
Accumulated other comprehensive loss | (468) | (451) |
Accumulated deficit | (325,848) | (296,221) |
Total stockholders' equity | 146,300 | 167,825 |
Total liabilities and stockholders' equity | $ 252,465 | $ 268,804 |
CONDENSED CONSOLIDATED BALANCE3
CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) (Parenthetical) - $ / shares | Mar. 31, 2018 | Dec. 31, 2017 |
Stockholders' equity: | ||
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized (in shares) | 100,000,000 | 100,000,000 |
Common stock, shares issued (in shares) | 66,803,803 | 66,541,629 |
Common stock, shares outstanding (in shares) | 66,803,803 | 66,541,629 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2018 | Mar. 31, 2017 | |
Revenue: | ||
Revenue | $ 17,108 | $ 6,094 |
Expenses: | ||
Research and development | 27,970 | 64,794 |
General and administrative | 19,465 | 4,676 |
Total operating expenses | 47,435 | 69,470 |
Loss from operations | (30,327) | (63,376) |
Other income (expense): | ||
Investment income | 868 | 61 |
Interest expense | 0 | (541) |
Other expense | (168) | 0 |
Loss before income tax expense | (29,627) | (63,856) |
Income tax expense | 0 | 0 |
Net loss | (29,627) | (63,856) |
Preferred Stock [Member] | ||
Other income (expense): | ||
Net loss | $ 0 | $ (63,856) |
Net loss per share, basic and diluted (in dollars per share) | $ 0 | $ (2.21) |
Weighted-average shares outstanding, basic and diluted (in shares) | 0 | 28,884,540 |
Common Stock [Member] | ||
Other income (expense): | ||
Net loss | $ (29,627) | $ 0 |
Net loss per share, basic and diluted (in dollars per share) | $ (0.44) | $ 0 |
Weighted-average shares outstanding, basic and diluted (in shares) | 66,616,337 | 0 |
Research and Development Under Collaborative Agreement [Member] | ||
Revenue: | ||
Revenue | $ 17,108 | $ 6,094 |
CONDENSED CONSOLIDATED STATEME5
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2018 | Mar. 31, 2017 | |
CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS [Abstract] | ||
Net loss | $ (29,627) | $ (63,856) |
Unrealized gains (losses) on investments, net of tax | (45) | (28) |
Currency translation adjustment | 28 | 6 |
Comprehensive loss | $ (29,644) | $ (63,878) |
CONDENSED CONSOLIDATED STATEME6
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2018 | Mar. 31, 2017 | |
Operating activities: | ||
Net loss | $ (29,627) | $ (63,856) |
Adjustments to reconcile net loss to net cash (used in) provided by operating activities: | ||
Depreciation | 27 | 29 |
Amortization of licenses | 30 | 30 |
Amortization of premium on investments, net | 149 | 24 |
Non-cash interest expense for line of credit with Ionis Pharmaceuticals, Inc. | 0 | 541 |
Stock-based compensation expense | 6,384 | 3,180 |
Changes in operating assets and liabilities: | ||
Contracts receivable | 5,413 | 0 |
Other current and long-term assets | (4,326) | (1,915) |
Payable to Ionis Pharmaceuticals, Inc. | 0 | (3,005) |
Accounts payable | 197 | (459) |
Payable to Ionis Pharmaceuticals, Inc. | 13,372 | (9,355) |
Accrued compensation | (310) | (1,658) |
Deferred rent | (10) | (9) |
Accrued liabilities | 5,843 | 331 |
Income taxes payable | 63 | (9) |
Deferred revenue | (13,968) | 102,301 |
Net cash (used in) provided by operating activities | (16,763) | 26,170 |
Investing activities: | ||
Purchases of short-term investments | (9,906) | (49,465) |
Proceeds from sale of short-term investments | 36,447 | 2,750 |
Net cash (used in) provided by investing activities | 26,541 | (46,715) |
Financing activities: | ||
Proceeds from exercise of common stock options and employee stock purchase plan issuances | 1,724 | 0 |
Proceeds from line of credit from Ionis Pharmaceuticals, Inc. | 0 | 91,000 |
Offering costs paid | 0 | (459) |
Net cash provided by financing activities | 1,724 | 90,541 |
Effect of exchange rates on cash | 38 | 5 |
Net increase in cash and cash equivalents | 11,540 | 70,001 |
Cash and cash equivalents at beginning of period | 58,367 | 7,857 |
Cash and cash equivalents at end of period | 69,907 | 77,858 |
Supplemental disclosures of non-cash financing activities: | ||
Unpaid deferred offering costs | $ 450 | $ 319 |
Basis of Presentation and Organ
Basis of Presentation and Organization | 3 Months Ended |
Mar. 31, 2018 | |
Basis of Presentation and Organization [Abstract] | |
Basis of Presentation and Organization | 1. Basis of Presentation and Organization The accompanying condensed consolidated financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the United States of America, or U.S. GAAP. Certain amounts in the prior period financial statements have been revised to conform to the presentation of the current period financial statements. See Note 2, Summary of Significant Accounting Policies The condensed consolidated financial statements include the accounts of Akcea Therapeutics, Inc. ("we," "our," and "us") and our wholly owned subsidiaries. All intercompany transactions and balances were eliminated in consolidation. We included all normal recurring adjustments in the financial statements which we considered necessary for a fair presentation of our financial position and our operating results and cash flows for the interim periods ended March 31, 2018 and 2017. Results for the interim periods are not necessarily indicative of the results for the entire year. For more complete financial information, these financial statements, and notes thereto, should be read in conjunction with the audited financial statements included on the Annual Report on Form 10-K for the fiscal year ended December 31, 2017. We were incorporated in Delaware in December 2014. We were organized by Ionis Pharmaceuticals, Inc., or Ionis, to . On July 19, 2017, we completed our initial public offering, or IPO. As of March 31, 2018, Ionis owned approximately 68% of our common stock and is our majority shareholder. Prior to our IPO, we were wholly owned by Ionis. In accordance with Accounting Standard Codification, or ASC, 205-40, Going Concern Subsequent Events |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2018 | |
Summary of Significant Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies The accounting policies followed in the preparation of the interim condensed consolidated financial statements are consistent in all material respects with those presented in Note 1 to our financial statements included on the Annual Report on Form 10-K for the year ended December 31, 2017 except as noted below with respect to our revenue recognition accounting policy. Use of Estimates The preparation of condensed consolidated financial statements in conformity with U.S. GAAP requires our management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. Actual results could differ from those estimates. Translation of Foreign Currency For our foreign subsidiaries that report in a functional currency other than U.S. dollars, we translate their assets and liabilities into U.S. dollars using the exchange rate at the balance sheet date. We translate revenue and expenses at the monthly average exchange rates for the period. We translate transactions in our capital accounts at the historic exchange rate in effect at the date of the transaction. We include foreign currency translation adjustments as a component of accumulated other comprehensive loss within the condensed consolidated statements of comprehensive loss. Revenue Recognition In May 2014, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, No. 2014-09, which amends the guidance for accounting for revenue from contracts with customers. This ASU supersedes the revenue recognition requirements in ASC Topic 605, Revenue Recognition Revenue from Contracts with Customers To determine revenue recognition for arrangements that an entity determines are within the scope of Topic 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of Topic 606, we assess the goods or services promised within each contract and determine those that are performance obligations, and assess whether each promised good or service is distinct. When we offer options for additional goods or services, such as Once performance obligations are identified, we then recognize as revenue the amount of the transaction price that we allocated to the respective performance obligation when (or as) We have one revenue stream from our strategic collaboration, option and license agreement, or collaboration agreement, with Novartis Pharma AG, or Novartis, which we entered into in January 2017. For a complete discussion of the accounting for our collaboration revenue, see Note 4, Strategic Collaboration with Novartis. Effective January 1, 2018, we adopted Topic 606 using the full retrospective transition method. Under this method, we revised our consolidated financial statements for prior period amounts including the interim periods included in this Report on Form 10-Q, as if Topic 606 had been effective for such periods. The references "as revised" used herein refer to revisions of data for the three months ended March 31, 2017 and the year ended December 31, 2017 as a result of our adoption of Topic 606. Impact of Adoption As a result of adopting Topic 606 on January 1, 2018, we have revised our comparative financial statements for the prior year as if Topic 606 had been effective for that period. Under Topic 605, we recognized revenue over time. Under Topic 606, we recognize revenue based on the input method based on total costs of performing services over time. As a result, the following financial statement line items for fiscal year 2017 were affected. Condensed Consolidated Balance Sheets December 31, 2017 (in thousands) As revised under Topic 606 As originally reported under Topic 605 Effect of change Current portion of deferred revenue $ 58,192 $ 50,579 $ 7,613 Long ‑ 12,501 8,306 4,195 Accumulated deficit $ (296,221 ) $ (284,413 ) $ (11,808 ) Condensed Consolidated Statements of Operations and Comprehensive Loss Three Months Ended March 31, 2017 (in thousands, except per share data) As revised under Topic 606 As originally reported under Topic 605 Effect of change Research and development revenue under collaborative agreements $ 6,094 $ 9,597 $ (3,503 ) Loss from operations (63,376 ) (59,873 ) (3,503 ) Net loss (63,856 ) (60,353 ) (3,503 ) Net loss per share of preferred stock, basic and diluted $ (2.21 ) $ (2.09 ) $ (0.12 ) Condensed Consolidated Statement of Cash Flows Three Months Ended March 31, 2017 (in thousands) As revised under Topic 606 As originally reported under Topic 605 Effect of change Net loss $ (63,856 ) $ (60,353 ) $ (3,503 ) Adjustments to reconcile net loss to net cash provided by operating activities: Deferred revenue 102,301 98,798 3,503 Cash and cash equivalents at beginning of period 7,857 7,857 — Cash and cash equivalents at end of period $ 77,858 $ 77,858 $ — New Accounting Pronouncements - Recently Issued In February 2016, the FASB issued amended accounting guidance related to lease accounting, which requires us to record all leases with a term longer than one year on our balance sheet. When we record leases on our balance sheet under the new guidance, we will record a liability with a value equal to the present value of payments we will make over the life of the lease and an asset representing the underlying leased asset. The new accounting guidance requires us to determine if any lease we have is an operating or financing lease, similar to current accounting guidance. We will record expense for an operating type lease on a straight-line basis as an operating expense and we will record expense for a financing type lease as interest expense. The new lease standard is effective for annual and interim periods beginning after December 15, 2018, with early adoption permitted. We must adopt the new standard on a modified retrospective basis, which requires us to reflect any leases we have on our consolidated balance sheet for the earliest comparative period presented. We are currently assessing the impact that adoption of this guidance will have on our consolidated financial statements and disclosures. In June 2016, the FASB issued guidance that changes the measurement of credit losses for most financial assets and certain other instruments. If we have credit losses, this updated guidance requires us to record allowances for these instruments under a new expected credit loss model. This model requires us to estimate the expected credit loss of an instrument over its lifetime, which represents the portion of the amortized cost basis that we do not expect to collect. This change will result in us remeasuring our allowance in each reporting period we have credit losses. The new standard is effective for annual and interim periods beginning after December 15, 2019. Early adoption is permitted for periods beginning after December 15, 2018. When we adopt the new standard, we will make any adjustments to beginning balances through a cumulative-effect adjustment to accumulated deficit on that date. We are currently assessing the timing of adoption as well as the effects it will have on our consolidated financial statements and disclosures. In February 2018, the FASB issued updated guidance for reclassification of tax effects from accumulated other comprehensive income (loss). The updated guidance gives entities an option to reclassify the stranded tax effects resulting from changes due to the Tax Act from accumulated other comprehensive income (loss) to accumulated deficit. The updated guidance is effective for all entities for fiscal years beginning after December 31, 2018, and interim periods within those fiscal years. Early adoption is permitted, and adoption is optional. We are currently assessing the effects this updated guidance could have on our consolidated financial statements and the timing of potential adoption. |
Investments and Fair Value Meas
Investments and Fair Value Measurements | 3 Months Ended |
Mar. 31, 2018 | |
Investments and Fair Value Measurements [Abstract] | |
Investments and Fair Value Measurements | 3. Investments and Fair Value Measurements Investments The following is a summary of our investments at March 31, 2018 and December 31, 2017 (in thousands): Gross Unrealized Estimated March 31, 2018 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 101,180 $ — $ (220 ) $ 100,960 Debt securities issued by U.S. government agencies 68,782 — (135 ) 68,647 Total securities with a maturity of one year or less 169,962 — (355 ) 169,607 Corporate debt securities 2,919 — (23 ) 2,896 Debt securities issued by U.S. government agencies 2,529 — (4 ) 2,525 Total securities with a maturity of one to two years 5,448 — (27 ) 5,421 Total available-for-sale securities $ 175,410 $ — $ (382 ) $ 175,028 Gross Unrealized Estimated December 31, 2017 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 132,434 $ — $ (206 ) $ 132,228 Debt securities issued by U.S. government agencies 38,135 — (59 ) 38,076 Total securities with a maturity of one year or less 170,569 — (265 ) 170,304 Corporate debt securities 8,267 — (35 ) 8,232 Debt securities issued by U.S. government agencies 23,264 — (37 ) 23,227 Total securities with a maturity of one to two years 31,531 — (72 ) 31,459 Total available-for-sale securities $ 202,100 $ — $ (337 ) $ 201,763 We recorded unrealized losses related to the securities listed above as of March 31, 2018 and December 31, 2017. We believe that the decline in value of these securities is temporary and primarily related to the change in market interest rates since purchase. We believe it is more likely than not that we will be able to hold our debt securities to maturity. Therefore, we anticipate a full recovery of our debt securities' amortized cost basis at maturity. All of our available-for-sale securities are available to us for use in our current operations. As a result, we categorized all of these securities as current assets even though the stated maturity of some individual securities may be one year or more beyond the balance sheet date. Fair Value Measurements We use a three-tier fair value hierarchy to prioritize the inputs used in our fair value measurements. These tiers include: Level 1, defined as observable inputs such as quoted prices in active markets for identical assets, which includes our money market funds and treasury securities classified as available-for-sale securities; Level 2, defined as inputs other than quoted prices in active markets that are either directly or indirectly observable, which includes our fixed income securities and commercial paper classified as available-for-sale securities; and Level 3, defined as unobservable inputs in which little or no market data exists, therefore requiring us to develop our own assumptions. We have not historically held any Level 3 investments. We recognize transfers between levels of the fair value hierarchy on the date of the event or change in circumstances that caused the transfer . The following tables present the major security types we held at March 31, 2018 and December 31, 2017 that are regularly measured and carried at fair value. The table segregates each security by the level within the fair value hierarchy of the valuation techniques we utilized to determine the respective securities' fair value (in thousands): At March 31, 2018 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Cash equivalents (1) $ 65,963 $ 65,963 $ — Corporate debt securities (2) 103,856 — 103,856 Debt securities issued by U.S. government agencies (2) 71,172 — 71,172 Total $ 240,991 $ 65,963 $ 175,028 At December 31, 2017 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Cash equivalents (1) $ 48,430 $ 48,430 $ — Corporate debt securities (2) 140,460 — 140,460 Debt securities issued by U.S. government agencies (2) 61,303 — 61,303 Total $ 250,193 $ 48,430 $ 201,763 (1) Included in cash and cash equivalents on our condensed consolidated balance sheets. (2) Included in short-term investments on our condensed consolidated balance sheets. We did not have any Level 3 investments at March 31, 2018 December 31, 2017 March 31, 2018 December 31, 2017 |
Strategic Collaboration with No
Strategic Collaboration with Novartis | 3 Months Ended |
Mar. 31, 2018 | |
Strategic Collaboration with Novartis [Abstract] | |
Strategic Collaboration with Novartis | 4. Strategic Collaboration with Novartis In January 2017, we initiated a strategic collaboration with Novartis for the development and commercialization of AKCEA-APO(a)-L Rx Rx We received a $75.0 million upfront payment in the first quarter of 2017, of which we retained $60.0 million and we paid Ionis $15.0 million as a sublicense fee under our license agreement with Ionis. If Novartis exercises its option for a drug, Novartis will pay us a license fee equal to $150.0 million for each drug licensed by Novartis. In addition, for AKCEA-APO(a)-L Rx e are eligible to receive up to $600.0 million in milestone payments, including $25.0 million for the achievement of a development milestone, up to $290.0 million for the achievement of regulatory milestones and up to $285.0 million for the achievement of commercialization milestones. In addition, for AKCEA-APOCIII-L Rx , we are eligible to receive up to $530.0 million in milestone payments, including $25.0 million for the achievement of a development milestone, up to $240.0 million for the achievement of regulatory milestones and up to $265.0 million for the achievement of commercialization milestones. We will earn the next milestone payment of $25.0 million under this collaboration if Novartis advances the Phase 3 study for either drug. We are also eligible to receive tiered royalties in the mid-teens to low twenty percent range on net sales of AKCEA-APO(a)-L Rx and AKCEA-APOCIII-L Rx . Novartis will reduce these royalties upon the expiration of certain patents or if a generic competitor negatively impacts the product in a specific country. We will pay 50% of these license fees, milestone payments and royalties to Ionis as a sublicense fee. We plan to co-commercialize any licensed drug commercialized by Novartis in selected markets under terms and conditions that we plan to negotiate with Novartis in the future, through the specialized sales force we are building to commercialize inotersen and volanesorsen. At commencement of our strategic collaboration, we identified the following four distinct performance obligations: ● Development activities for AKCEA-APO(a)-L Rx ● Development activities for AKCEA-APOCIII-L Rx ● API for AKCEA-APO(a)-L Rx ● API for AKCEA-APOCIII-L Rx The development activities and the supply of API are distinct because Novartis or another third party could provide these items without our assistance. We determined the transaction price for the Novartis collaboration was $108.4 million, comprised of the following: ● $75.0 million from the upfront payment we received; ● $28.4 million for the premium paid by Novartis, which represents the excess of the fair value Ionis received from Novartis' purchase of Ionis' stock at a premium in the first quarter of 2017; and ● $5.0 million for the premium Novartis would have paid to purchase Ionis' stock if we did not complete our IPO within 15 months of the inception of the agreement. We are recognizing the $75.0 million upfront payment plus the premium paid by Novartis from its purchase of Ionis' stock and the premium associated with Novartis' obligation to purchase Ionis' stock if we did not complete our IPO because we are the party providing the services and API under the collaboration agreement. None of the development or regulatory milestone payments have been included in the transaction price, as all milestone payments are fully constrained. As part of our evaluation of the constraint, we considered numerous factors, including the fact that achievement of the milestones is outside of our control and contingent upon the success of our clinical trials, Novartis' efforts, and the receipt of regulatory approval. We will re-evaluate the transaction price, including estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur. Based on the distinct performance obligations under the Novartis collaboration, we allocated the $108.4 million transaction price based on relative stand-alone selling prices of each of our performance obligations as follows: ● $64.0 million for development services for AKCEA-APO(a)-L Rx ● $40.1 million for development services for AKCEA-APOCIII-L Rx ● $1.5 million for the delivery of AKCEA-APO(a)-L Rx ● $2.8 million for the delivery of AKCEA-APOCIII-L Rx We are recognizing revenue related to each of our performance obligations as follows: ● We will satisfy the development services performance obligation for AKCEA-APO(a)-L Rx ● We will satisfy the development services performance obligation for AKCEA-APOCIII-L Rx ● We will recognize the amount attributed to the AKCEA-APO(a)-L Rx ● We recognized the amount attributed to the AKCEA-APOCIII-L Rx At March 31, 2018, the aggregate transaction price allocated to our remaining performance obligations was $56.7 million, which we are recognizing over the estimated period of our performance obligation. Additionally, we and Ionis entered into a stock purchase agreement, or SPA, with Novartis. Under the SPA, in July 2017, Novartis purchased $50.0 million of our common stock in a separate private placement concurrent with the completion of our IPO at a price per share equal to the IPO price. Our IPO is discussed in Note 9, Initial Public Offering During the three months ended March 31, 2018, we earned revenue of $17.1 million from our relationship with Novartis, representing 100% of our revenue. During the three months ended March 31, 2018, we recognized $17.1 million of revenue from amounts that were in our beginning deferred revenue balance. Our consolidated balance sheet at March 31, 2018 and December 31, 2017 (as revised) included deferred revenue of $56.7 million and $70.7 million, respectively, related to our relationship with Novartis. |
Development, Commercialization
Development, Commercialization and License Agreement and Services Agreement with Ionis | 3 Months Ended |
Mar. 31, 2018 | |
Development, Commercialization and License Agreement and Services Agreement with Ionis [Abstract] | |
Development, Commercialization and License Agreement and Services Agreement with Ionis | 5. Development, Commercialization and License Agreement and Services Agreement with Ionis We entered into a development, commercialization and license agreement and a services agreement in December 2015 with Ionis. The following section summarizes these related party agreements with Ionis. Development, Commercialization and License Agreement Our development, commercialization and license agreement, or the license agreement, with Ionis granted exclusive rights to us to develop and commercialize volanesorsen, AKCEA-APO(a)-L Rx Rx Rx We and Ionis share development responsibilities for the Lipid Drugs. We pay Ionis for the research and development expenses it incurs on our behalf, which include both external and internal expenses. External research and development expenses include costs for contract research organizations, or CROs, costs to conduct nonclinical and clinical studies on our drugs, costs to acquire and evaluate clinical study data, such as investigator grants, patient screening fees and laboratory work, and fees paid to consultants. Internal research and development expenses include costs for the work that Ionis' research and development employees perform for us. Ionis charges us a full-time equivalent rate that covers personnel-related expenses, including salaries and benefits, plus an allocation of facility-related expenses, including rent, utilities, insurance and property taxes, for those development employees who work either directly or indirectly on the development of our drugs. We also pay Ionis for the API, and drug product we use in our nonclinical and clinical studies for all of our drugs. Ionis manufactures the API for us and charges us a price per gram consistent with the price Ionis charges its pharmaceutical partners, which includes the cost for direct materials, direct labor and overhead required to manufacture the API. If we need the API filled in vials for our clinical studies and Ionis contracts with a third party to perform this work, Ionis will charge us for the resulting cost. As we commercialize each of the Lipid Drugs, we will pay Ionis royalties from the mid-teens to the mid-twenty percent range on sales related to the Lipid Drugs that we sell. If we sell a Lipid Drug for a Rare Disease Indication (defined in the agreement as less than 500,000 patients worldwide or an indication that required a Phase 3 program of less than 1,000 patients and less than two years of treatment), we will pay a higher royalty rate to Ionis than if we sell a Lipid Drug for a Broad Disease Patient Population (defined in the agreement as more than 500,000 patients worldwide or an indication that required a Phase 3 program of 1,000 or more patients and two or more years of treatment). Other than with respect to the drugs licensed to Novartis under the collaboration agreement, if our annual sales reach $500.0 million, $1.0 billion and $2.0 billion, we will be obligated to pay Ionis sales milestones in the amount of $50.0 million for each sales milestone reached by each Lipid Drug. If and when triggered, we will pay Ionis each of these sales milestones over the subsequent 12 quarters in equal payments. We may terminate this agreement if Ionis is in material breach of the agreement. Ionis may terminate this agreement if we are in material breach of the agreement. In each circumstance the party that is in breach will have an opportunity to cure the breach prior to the other party terminating this agreement. In the first quarter of 2017, we entered into letter agreements with Ionis to reflect the agreed upon payment terms with respect to the upfront option payment that we received from Novartis and to allocate the premium that Novartis paid for Ionis' common stock in connection with our strategic collaboration with Novartis. For additional detail regarding our strategic collaboration with Novartis, see Note 4, Strategic Collaboration with Novartis. Services Agreement Our services agreement with Ionis is designed to be flexible to adjust for our increasing capabilities in various functions. Under the services agreement, Ionis provides us certain services, including, without limitation, general and administrative support services and development support services. Ionis allocated a certain percentage of personnel to perform the services that it provides to us based on its good faith estimate of the required services. We pay Ionis for these allocated costs, which reflect the Ionis full-time equivalent, or FTE, rate for the applicable personnel, plus out-of-pocket expenses such as occupancy costs associated with the FTEs allocated to providing us these services. We do not pay a mark-up or profit on the external or internal expenses Ionis bills to us. Ionis invoices us quarterly for all amounts due under the services agreement and payments are due within 30 days of the receipt of an invoice. In addition, as long as Ionis continues to consolidate our financials, we will comply with Ionis' policies and procedures and internal controls. As long as we are consolidated into Ionis' financial statements under U.S. GAAP, we may continue to access the following services from Ionis: ● investor relations services, ● human resources and personnel services, ● risk management and insurance services, ● tax related services, ● corporate record keeping services, ● financial and accounting services, ● credit services, and ● COO/CFO/CBO oversight. However, if we wanted to provide for our own human resources and personnel services, and doing so would not negatively impact Ionis' internal controls and procedures for financial reporting, we can negotiate in good faith with Ionis for a reduced scope of services related to human resources and personnel services. When Ionis determines it should no longer consolidate our financials, we may mutually agree with Ionis in writing to extend the term of this arrangement in six-month increments. We can establish our own benefits programs or continue to use Ionis' benefits, however we must provide Ionis a minimum advance notice to opt-out of using Ionis' benefits. We do not currently plan to establish our own benefits programs at this time or in the near future. As of March 31, 2018 and December 31, 2017, we owed Ionis $27.7 million and $14.4 million, respectively. The following table summarizes the amounts included in our operating expenses that were generated by transactions with Ionis for the following periods (in thousands): Three Months Ended March 31, 2018 2017 Services performed by Ionis $ 1,945 $ 2,957 Active pharmaceutical ingredient manufactured by Ionis 5,229 3,083 Sublicensing expenses — 48,394 Out-of-pocket expenses paid by Ionis 6,236 8,868 Total expenses generated by transactions with Ionis 13,410 63,302 Payable balance to Ionis at the beginning of the period 14,365 24,355 Prepaid amounts to Ionis — 3,005 Less: total amounts paid to Ionis during the period (38 ) (42,268 ) Less: non-cash sublicensing expenses — (33,394 ) Total amount payable to Ionis at period end $ 27,737 $ 15,000 |
Stock-Based Compensation
Stock-Based Compensation | 3 Months Ended |
Mar. 31, 2018 | |
Stock-Based Compensation [Abstract] | |
Stock-Based Compensation | 6. Stock-Based Compensation Stock Plans 2015 Equity Incentive Plan In December 2015, our board of directors and stockholder adopted and approved our 2015 Equity Incentive Plan, or the 2015 Plan. In May 2017 and June 2017, our board of directors and stockholder, respectively, approved an amendment to our 2015 Equity Incentive Plan in order to, among other things, increase the number of shares of common stock reserved for issuance thereunder to 8,500,000 shares of common stock in conjunction with the IPO. In December 2017 and April 2018, our board of directors and our majority stockholder, respectively, approved an additional amendment to our 2015 Equity Incentive Plan to increase the number of shares of common stock reserved for issuance thereunder to 13,500,000 shares of common stock. As of March 31, 2018, the aggregate number of shares of common stock that may be issued pursuant to stock awards under the 2015 Plan was 8,500,000 shares, not including an additional 5,000,000 shares approved by the Board of Directors in December 2017, which are subject to shareholder approval on June 1, 2018. The 2015 Plan also provides for the grant of non-statutory stock options, or NSOs, incentive stock options, or ISOs, stock appreciation rights, restricted stock awards and restricted stock unit awards. At March 31, 2018, assuming approval of the additional 5,000,000 shares above, a total of 9,298,024 options were outstanding, of which 3,276,937 were exercisable, 32,529 restricted stock unit awards were outstanding, and 4,201,976 shares were available for future grant under the 2015 Plan. 2017 Employee Stock Purchase Plan In May 2017 and June 2017, our board of directors and stockholder, respectively, approved our 2017 Employee Stock Purchase Plan, or 2017 ESPP, which became effective upon the completion of our IPO, and the reservation for issuance thereunder of 1,165,416 shares of common stock. In addition, the number of shares of common stock that may be issued under the ESPP will automatically increase commencing on January 1, 2018 and ending on (and including) January 1, 2027 in an amount equal to the lesser of (i) 1% of the total number of shares of Common Stock outstanding on December 31st of the preceding calendar year, and (ii) 500,000 shares of Common Stock. During the three months ended March 31, 2018, 15,732 shares were issued under our 2017 ESPP. As of March 31, 2018, the aggregate number of shares of common stock that may be issued pursuant to the 2017 ESPP was 1,149,684 shares. At March 31, 2018, accrued liabilities included $144,000 of ESPP contributions related to our current enrollment period for which the related shares will be issued on July 2, 2018. Stock-Based Compensation The following table summarizes stock-based compensation expense for the three months ended March 31, 2018 and 2017 (in thousands): Three Months Ended March 31, 2018 2017 Research and development expenses $ 2,314 $ 1,600 General and administrative expenses 4,070 1,580 Total $ 6,384 $ 3,180 |
Accumulated Other Comprehensive
Accumulated Other Comprehensive Loss | 3 Months Ended |
Mar. 31, 2018 | |
Accumulated Other Comprehensive Loss [Abstract] | |
Accumulated Other Comprehensive Loss | 7. Accumulated Other Comprehensive Loss The following table summarizes changes in accumulated other comprehensive loss (in thousands): 2018 Balance, as of December 31, 2017 $ (451 ) Unrealized gains (losses) on investments, net of tax (1) (45 ) Currency translation adjustment 28 Net other comprehensive income (loss) (17 ) Balance, as of March 31, 2018 $ (468 ) (1) There was no tax benefit for other comprehensive income (loss) for the three months ended March 31, 2018. |
Basic and Diluted Net Loss Per
Basic and Diluted Net Loss Per Share | 3 Months Ended |
Mar. 31, 2018 | |
Basic and Diluted Net Loss Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | 8. Basic and Diluted Net Loss Per Share The following table summarizes the calculation of basic EPS for the three months ended March 31, 2018 and 2017 (in thousands, except share and per share amounts): Three Months Ended March 31, 2018 2017 Losses allocated to preferred shares $ — $ (63,856 ) Weighted-average preferred shares outstanding — 28,884,540 Basic loss per preferred share $ — $ (2.21 ) Losses allocated to common shares $ (29,627 ) $ — Weighted-average common shares outstanding 66,616,337 — Basic loss per common share $ (0.44 ) $ — For the three months ended March 31, 2018 and 2017, we incurred a net loss; therefore, we did not include dilutive common equivalent shares in the computation of diluted net loss per share because the effect would have been anti-dilutive. Common stock from the following would have had an anti-dilutive effect on net loss per share: ● Options to purchase common stock; ● Unvested restricted stock units; and ● Employee Stock Purchase Plan, or ESPP. |
Initial Public Offering
Initial Public Offering | 3 Months Ended |
Mar. 31, 2018 | |
Initial Public Offering [Abstract] | |
Initial Public Offering | 9. Initial Public Offering On July 19, 2017, we completed our IPO. Total net proceeds were $182.3 million, including the following: ● $132.3 million from the sale of 17,968,750 shares of our common stock in our IPO of which $25 million was invested by Ionis; and ● $50.0 million from the purchase of 6,250,000 shares by Novartis in a concurrent private placement. In addition, both of the following occurred in connection with the completion of our IPO on July 19, 2017: ● the conversion of all outstanding shares of Series A convertible preferred stock into 28,884,540 shares of our common stock; and ● the conversion of $106.0 million of outstanding principal plus accrued interest from the line of credit into 13,438,339 shares of common stock. |
Subsequent Events
Subsequent Events | 3 Months Ended |
Mar. 31, 2018 | |
Subsequent Events [Abstract] | |
Subsequent Events | 10. Subsequent Events Operating Lease On April 5, 2018, we entered into an operating lease agreement with MEPT Seaport 13 Stillings LLC, or MEPT, for 30,175 square feet of office space located in Boston, Massachusetts for our new corporate headquarters. The anticipated commencement date of the lease is August 15, 2018. The initial term of the lease is 123 months with one five-year renewal option. Future minimum annual lease payments under this lease are $0.8 million in 2018, $2.2 million in 2019, $2.3 million in 2020, $2.3 million in 2021, $2.3 million in 2022, and $14.2 million thereafter. MEPT will provide us with a three-month free rent period and a tenant improvement allowance up to $3.8 million. Inotersen Development, Commercialization, Collaboration and License Agreement On April 17, 2018, our stockholders, other than Ionis and its affiliates, approved the development, commercialization, collaboration and license agreement, or License Agreement, and a stock purchase agreement, or Ionis SPA, with Ionis, our majority shareholder which was entered into on March 14, 2018. In addition, in connection with these agreements, we entered into an amended and restated services agreement, or Amended Services Agreement, and an amended and restated investor rights agreement, or Amended Investor Rights Agreement, with Ionis. We determined that the License Agreement and Ionis SPA included provisions which required the approval of the agreements by our stockholders, other than Ionis and its affiliates, which we deemed was not perfunctory in nature, therefore, we concluded that the approved date of the agreements for accounting purposes would be April 17, 2018, the date on which such approval was received. In accordance with the terms and provisions of the License Agreement, we received rights to: ● commercialize inotersen following receipt of regulatory approval and perform certain other non-commercial activities with respect to inotersen, in each case, in accordance with a global strategic plan; ● partner on the completion of all pivotal studies, of a follow-on drug to inotersen, AKCEA-TTR-L Rx and perform other non-commercial activities with respect to AKCEA-TTR-L Rx ; ● commercialize AKCEA-TTR-L Rx , following receipt of regulatory approval in accordance with a global strategic plan; ● share in profits and losses with respect to inotersen and AKCEA-TTR-L Rx ; ● manufacture (including through a third party) each product following receipt of regulatory approval for such product; and ● sublicense the development and commercialization of either product to third parties or affiliates, with the consent of Ionis. As payment for the grant of rights under the License Agreement, we paid an upfront licensing fee of $150.0 million, which was paid through the issuance of 8 million shares of our common stock priced by reference to a recent trading average. In addition, we will be obligated to make milestone payments to Ionis in connection with the achievement of certain development, regulatory and commercialization events. These milestone payments include up to $110.0 million, if all inotersen approval milestones are met; up to $145.0 million, if all AKCEA-TTR-L Rx We and Ionis also agreed to share inotersen and AKCEA-TTR-L Rx Rx Rx Rx The License Agreement will remain in effect until the expiration of all included payment obligations, or unless earlier terminated. The License Agreement can be terminated by mutual consent of us and Ionis, by either us or Ionis upon certain events, by either party upon material breach, or by Akcea for convenience upon providing 90 days written notice to Ionis. Upon termination all rights received under the License Agreement will terminate. To support the commercialization of inotersen and AKCEA-TTR-L Rx The Amended Services Agreement allows for the expansion of general and administrative services provided to us by Ionis to cover the inotersen and AKCEA-TTR-L Rx In connection with the licensing transaction, we amended our Certificate of Incorporation to increase the authorized shares of common stock from 100,000,000 shares to 125,000,000 shares. |
Basis of Presentation and Org17
Basis of Presentation and Organization (Policies) | 3 Months Ended |
Mar. 31, 2018 | |
Basis of Presentation and Organization [Abstract] | |
Basis of Presentation | The accompanying condensed consolidated financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the United States of America, or U.S. GAAP. Certain amounts in the prior period financial statements have been revised to conform to the presentation of the current period financial statements. See Note 2, Summary of Significant Accounting Policies |
Consolidation | The condensed consolidated financial statements include the accounts of Akcea Therapeutics, Inc. ("we," "our," and "us") and our wholly owned subsidiaries. All intercompany transactions and balances were eliminated in consolidation. We included all normal recurring adjustments in the financial statements which we considered necessary for a fair presentation of our financial position and our operating results and cash flows for the interim periods ended March 31, 2018 and 2017. Results for the interim periods are not necessarily indicative of the results for the entire year. For more complete financial information, these financial statements, and notes thereto, should be read in conjunction with the audited financial statements included on the Annual Report on Form 10-K for the fiscal year ended December 31, 2017. |
Summary of Significant Accoun18
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2018 | |
Summary of Significant Accounting Policies [Abstract] | |
Use of Estimates | Use of Estimates The preparation of condensed consolidated financial statements in conformity with U.S. GAAP requires our management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. Actual results could differ from those estimates. |
Translation of Foreign Currency | Translation of Foreign Currency For our foreign subsidiaries that report in a functional currency other than U.S. dollars, we translate their assets and liabilities into U.S. dollars using the exchange rate at the balance sheet date. We translate revenue and expenses at the monthly average exchange rates for the period. We translate transactions in our capital accounts at the historic exchange rate in effect at the date of the transaction. We include foreign currency translation adjustments as a component of accumulated other comprehensive loss within the condensed consolidated statements of comprehensive loss. |
Revenue Recognition | Revenue Recognition In May 2014, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, No. 2014-09, which amends the guidance for accounting for revenue from contracts with customers. This ASU supersedes the revenue recognition requirements in ASC Topic 605, Revenue Recognition Revenue from Contracts with Customers To determine revenue recognition for arrangements that an entity determines are within the scope of Topic 606, the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. At contract inception, once the contract is determined to be within the scope of Topic 606, we assess the goods or services promised within each contract and determine those that are performance obligations, and assess whether each promised good or service is distinct. When we offer options for additional goods or services, such as Once performance obligations are identified, we then recognize as revenue the amount of the transaction price that we allocated to the respective performance obligation when (or as) We have one revenue stream from our strategic collaboration, option and license agreement, or collaboration agreement, with Novartis Pharma AG, or Novartis, which we entered into in January 2017. For a complete discussion of the accounting for our collaboration revenue, see Note 4, Strategic Collaboration with Novartis. Effective January 1, 2018, we adopted Topic 606 using the full retrospective transition method. Under this method, we revised our consolidated financial statements for prior period amounts including the interim periods included in this Report on Form 10-Q, as if Topic 606 had been effective for such periods. The references "as revised" used herein refer to revisions of data for the three months ended March 31, 2017 and the year ended December 31, 2017 as a result of our adoption of Topic 606. Impact of Adoption As a result of adopting Topic 606 on January 1, 2018, we have revised our comparative financial statements for the prior year as if Topic 606 had been effective for that period. Under Topic 605, we recognized revenue over time. Under Topic 606, we recognize revenue based on the input method based on total costs of performing services over time. As a result, the following financial statement line items for fiscal year 2017 were affected. Condensed Consolidated Balance Sheets December 31, 2017 (in thousands) As revised under Topic 606 As originally reported under Topic 605 Effect of change Current portion of deferred revenue $ 58,192 $ 50,579 $ 7,613 Long ‑ 12,501 8,306 4,195 Accumulated deficit $ (296,221 ) $ (284,413 ) $ (11,808 ) Condensed Consolidated Statements of Operations and Comprehensive Loss Three Months Ended March 31, 2017 (in thousands, except per share data) As revised under Topic 606 As originally reported under Topic 605 Effect of change Research and development revenue under collaborative agreements $ 6,094 $ 9,597 $ (3,503 ) Loss from operations (63,376 ) (59,873 ) (3,503 ) Net loss (63,856 ) (60,353 ) (3,503 ) Net loss per share of preferred stock, basic and diluted $ (2.21 ) $ (2.09 ) $ (0.12 ) Condensed Consolidated Statement of Cash Flows Three Months Ended March 31, 2017 (in thousands) As revised under Topic 606 As originally reported under Topic 605 Effect of change Net loss $ (63,856 ) $ (60,353 ) $ (3,503 ) Adjustments to reconcile net loss to net cash provided by operating activities: Deferred revenue 102,301 98,798 3,503 Cash and cash equivalents at beginning of period 7,857 7,857 — Cash and cash equivalents at end of period $ 77,858 $ 77,858 $ — |
New Accounting Pronouncements - Recently Issued | New Accounting Pronouncements - Recently Issued In February 2016, the FASB issued amended accounting guidance related to lease accounting, which requires us to record all leases with a term longer than one year on our balance sheet. When we record leases on our balance sheet under the new guidance, we will record a liability with a value equal to the present value of payments we will make over the life of the lease and an asset representing the underlying leased asset. The new accounting guidance requires us to determine if any lease we have is an operating or financing lease, similar to current accounting guidance. We will record expense for an operating type lease on a straight-line basis as an operating expense and we will record expense for a financing type lease as interest expense. The new lease standard is effective for annual and interim periods beginning after December 15, 2018, with early adoption permitted. We must adopt the new standard on a modified retrospective basis, which requires us to reflect any leases we have on our consolidated balance sheet for the earliest comparative period presented. We are currently assessing the impact that adoption of this guidance will have on our consolidated financial statements and disclosures. In June 2016, the FASB issued guidance that changes the measurement of credit losses for most financial assets and certain other instruments. If we have credit losses, this updated guidance requires us to record allowances for these instruments under a new expected credit loss model. This model requires us to estimate the expected credit loss of an instrument over its lifetime, which represents the portion of the amortized cost basis that we do not expect to collect. This change will result in us remeasuring our allowance in each reporting period we have credit losses. The new standard is effective for annual and interim periods beginning after December 15, 2019. Early adoption is permitted for periods beginning after December 15, 2018. When we adopt the new standard, we will make any adjustments to beginning balances through a cumulative-effect adjustment to accumulated deficit on that date. We are currently assessing the timing of adoption as well as the effects it will have on our consolidated financial statements and disclosures. In February 2018, the FASB issued updated guidance for reclassification of tax effects from accumulated other comprehensive income (loss). The updated guidance gives entities an option to reclassify the stranded tax effects resulting from changes due to the Tax Act from accumulated other comprehensive income (loss) to accumulated deficit. The updated guidance is effective for all entities for fiscal years beginning after December 31, 2018, and interim periods within those fiscal years. Early adoption is permitted, and adoption is optional. We are currently assessing the effects this updated guidance could have on our consolidated financial statements and the timing of potential adoption. |
Summary of Significant Accoun19
Summary of Significant Accounting Policies (Tables) | 3 Months Ended |
Mar. 31, 2018 | |
Summary of Significant Accounting Policies [Abstract] | |
Impact of Adoption of Topic 606 | Condensed Consolidated Balance Sheets December 31, 2017 (in thousands) As revised under Topic 606 As originally reported under Topic 605 Effect of change Current portion of deferred revenue $ 58,192 $ 50,579 $ 7,613 Long ‑ 12,501 8,306 4,195 Accumulated deficit $ (296,221 ) $ (284,413 ) $ (11,808 ) Condensed Consolidated Statements of Operations and Comprehensive Loss Three Months Ended March 31, 2017 (in thousands, except per share data) As revised under Topic 606 As originally reported under Topic 605 Effect of change Research and development revenue under collaborative agreements $ 6,094 $ 9,597 $ (3,503 ) Loss from operations (63,376 ) (59,873 ) (3,503 ) Net loss (63,856 ) (60,353 ) (3,503 ) Net loss per share of preferred stock, basic and diluted $ (2.21 ) $ (2.09 ) $ (0.12 ) Condensed Consolidated Statement of Cash Flows Three Months Ended March 31, 2017 (in thousands) As revised under Topic 606 As originally reported under Topic 605 Effect of change Net loss $ (63,856 ) $ (60,353 ) $ (3,503 ) Adjustments to reconcile net loss to net cash provided by operating activities: Deferred revenue 102,301 98,798 3,503 Cash and cash equivalents at beginning of period 7,857 7,857 — Cash and cash equivalents at end of period $ 77,858 $ 77,858 $ — |
Investments and Fair Value Me20
Investments and Fair Value Measurements (Tables) | 3 Months Ended |
Mar. 31, 2018 | |
Investments and Fair Value Measurements [Abstract] | |
Summary of Investments | The following is a summary of our investments at March 31, 2018 and December 31, 2017 (in thousands): Gross Unrealized Estimated March 31, 2018 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 101,180 $ — $ (220 ) $ 100,960 Debt securities issued by U.S. government agencies 68,782 — (135 ) 68,647 Total securities with a maturity of one year or less 169,962 — (355 ) 169,607 Corporate debt securities 2,919 — (23 ) 2,896 Debt securities issued by U.S. government agencies 2,529 — (4 ) 2,525 Total securities with a maturity of one to two years 5,448 — (27 ) 5,421 Total available-for-sale securities $ 175,410 $ — $ (382 ) $ 175,028 Gross Unrealized Estimated December 31, 2017 Cost Gains Losses Fair Value Available-for-sale securities: Corporate debt securities $ 132,434 $ — $ (206 ) $ 132,228 Debt securities issued by U.S. government agencies 38,135 — (59 ) 38,076 Total securities with a maturity of one year or less 170,569 — (265 ) 170,304 Corporate debt securities 8,267 — (35 ) 8,232 Debt securities issued by U.S. government agencies 23,264 — (37 ) 23,227 Total securities with a maturity of one to two years 31,531 — (72 ) 31,459 Total available-for-sale securities $ 202,100 $ — $ (337 ) $ 201,763 |
Assets Measured at Fair Value on a Recurring Basis | The following tables present the major security types we held at March 31, 2018 and December 31, 2017 that are regularly measured and carried at fair value. The table segregates each security by the level within the fair value hierarchy of the valuation techniques we utilized to determine the respective securities' fair value (in thousands): At March 31, 2018 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Cash equivalents (1) $ 65,963 $ 65,963 $ — Corporate debt securities (2) 103,856 — 103,856 Debt securities issued by U.S. government agencies (2) 71,172 — 71,172 Total $ 240,991 $ 65,963 $ 175,028 At December 31, 2017 Quoted Prices in Active Markets (Level 1) Significant Other Observable Inputs (Level 2) Cash equivalents (1) $ 48,430 $ 48,430 $ — Corporate debt securities (2) 140,460 — 140,460 Debt securities issued by U.S. government agencies (2) 61,303 — 61,303 Total $ 250,193 $ 48,430 $ 201,763 (1) Included in cash and cash equivalents on our condensed consolidated balance sheets. (2) Included in short-term investments on our condensed consolidated balance sheets. |
Development, Commercializatio21
Development, Commercialization and License Agreement and Services Agreement with Ionis (Tables) | 3 Months Ended |
Mar. 31, 2018 | |
Development, Commercialization and License Agreement and Services Agreement with Ionis [Abstract] | |
Operating Expenses Generated by Transactions with Ionis | The following table summarizes the amounts included in our operating expenses that were generated by transactions with Ionis for the following periods (in thousands): Three Months Ended March 31, 2018 2017 Services performed by Ionis $ 1,945 $ 2,957 Active pharmaceutical ingredient manufactured by Ionis 5,229 3,083 Sublicensing expenses — 48,394 Out-of-pocket expenses paid by Ionis 6,236 8,868 Total expenses generated by transactions with Ionis 13,410 63,302 Payable balance to Ionis at the beginning of the period 14,365 24,355 Prepaid amounts to Ionis — 3,005 Less: total amounts paid to Ionis during the period (38 ) (42,268 ) Less: non-cash sublicensing expenses — (33,394 ) Total amount payable to Ionis at period end $ 27,737 $ 15,000 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2018 | |
Stock-Based Compensation [Abstract] | |
Stock-Based Compensation Expense | The following table summarizes stock-based compensation expense for the three months ended March 31, 2018 and 2017 (in thousands): Three Months Ended March 31, 2018 2017 Research and development expenses $ 2,314 $ 1,600 General and administrative expenses 4,070 1,580 Total $ 6,384 $ 3,180 |
Accumulated Other Comprehensi23
Accumulated Other Comprehensive Loss (Tables) | 3 Months Ended |
Mar. 31, 2018 | |
Accumulated Other Comprehensive Loss [Abstract] | |
Changes in Accumulated Other Comprehensive Loss | The following table summarizes changes in accumulated other comprehensive loss (in thousands): 2018 Balance, as of December 31, 2017 $ (451 ) Unrealized gains (losses) on investments, net of tax (1) (45 ) Currency translation adjustment 28 Net other comprehensive income (loss) (17 ) Balance, as of March 31, 2018 $ (468 ) (1) There was no tax benefit for other comprehensive income (loss) for the three months ended March 31, 2018. |
Basic and Diluted Net Loss Pe24
Basic and Diluted Net Loss Per Share (Tables) | 3 Months Ended |
Mar. 31, 2018 | |
Basic and Diluted Net Loss Per Share [Abstract] | |
Basic Loss Per Share | The following table summarizes the calculation of basic EPS for the three months ended March 31, 2018 and 2017 (in thousands, except share and per share amounts): Three Months Ended March 31, 2018 2017 Losses allocated to preferred shares $ — $ (63,856 ) Weighted-average preferred shares outstanding — 28,884,540 Basic loss per preferred share $ — $ (2.21 ) Losses allocated to common shares $ (29,627 ) $ — Weighted-average common shares outstanding 66,616,337 — Basic loss per common share $ (0.44 ) $ — |
Basis of Presentation and Org25
Basis of Presentation and Organization (Details) - USD ($) $ in Thousands | Apr. 17, 2018 | Apr. 30, 2018 | Mar. 31, 2018 | Mar. 31, 2017 | Dec. 31, 2017 |
Basis of Presentation and Organization [Abstract] | |||||
Ownership percentage by Ionis | 68.00% | ||||
Accumulated deficit | $ (325,848) | $ (296,221) | |||
Net loss | (29,627) | $ (63,856) | |||
Net cash used in operating activities | (16,763) | $ 26,170 | |||
Cash, cash equivalents and short-term investments | $ 244,935 | ||||
Subsequent Event [Member] | |||||
Basis of Presentation and Organization [Abstract] | |||||
Ownership percentage by Ionis | 75.00% | ||||
Basis of Presentation and Organization [Abstract] | |||||
Proceeds from issuance of common stock to Ionis | $ 200,000 | $ 200,000 |
Summary of Significant Accoun26
Summary of Significant Accounting Policies (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | ||
Mar. 31, 2018 | Mar. 31, 2017 | Dec. 31, 2017 | |
Condensed Consolidated Balance Sheets [Abstract] | |||
Current portion of deferred revenue | $ 48,866 | $ 58,192 | |
Long-term portion of deferred revenue | 7,859 | 12,501 | |
Accumulated deficit | (325,848) | (296,221) | |
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||
Revenue | 17,108 | $ 6,094 | |
Loss from operations | (30,327) | (63,376) | |
Net loss | (29,627) | (63,856) | |
Condensed Consolidated Statement of Cash Flows [Abstract] | |||
Net loss | (29,627) | (63,856) | |
Adjustments to reconcile net loss to net cash (used in) provided by operating activities: | |||
Deferred revenue | (13,968) | 102,301 | |
Cash and cash equivalents at beginning of period | 58,367 | 7,857 | |
Cash and cash equivalents at end of period | 69,907 | 77,858 | |
Preferred Stock [Member] | |||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||
Net loss | $ 0 | $ (63,856) | |
Net loss per share, basic and diluted (in dollars per share) | $ 0 | $ (2.21) | |
Condensed Consolidated Statement of Cash Flows [Abstract] | |||
Net loss | $ 0 | $ (63,856) | |
Research and Development Under Collaborative Agreement [Member] | |||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||
Revenue | $ 17,108 | 6,094 | |
As Originally Reported Under Topic 605 [Member] | ASU 2014-09 [Member] | |||
Condensed Consolidated Balance Sheets [Abstract] | |||
Current portion of deferred revenue | 50,579 | ||
Long-term portion of deferred revenue | 8,306 | ||
Accumulated deficit | (284,413) | ||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||
Loss from operations | (59,873) | ||
Net loss | (60,353) | ||
Condensed Consolidated Statement of Cash Flows [Abstract] | |||
Net loss | (60,353) | ||
Adjustments to reconcile net loss to net cash (used in) provided by operating activities: | |||
Deferred revenue | 98,798 | ||
Cash and cash equivalents at beginning of period | 7,857 | ||
Cash and cash equivalents at end of period | $ 77,858 | ||
As Originally Reported Under Topic 605 [Member] | ASU 2014-09 [Member] | Preferred Stock [Member] | |||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||
Net loss per share, basic and diluted (in dollars per share) | $ (2.09) | ||
As Originally Reported Under Topic 605 [Member] | ASU 2014-09 [Member] | Research and Development Under Collaborative Agreement [Member] | |||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||
Revenue | $ 9,597 | ||
Effect of Change [Member] | ASU 2014-09 [Member] | |||
Condensed Consolidated Balance Sheets [Abstract] | |||
Current portion of deferred revenue | 7,613 | ||
Long-term portion of deferred revenue | 4,195 | ||
Accumulated deficit | $ (11,808) | ||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||
Loss from operations | (3,503) | ||
Net loss | (3,503) | ||
Condensed Consolidated Statement of Cash Flows [Abstract] | |||
Net loss | (3,503) | ||
Adjustments to reconcile net loss to net cash (used in) provided by operating activities: | |||
Deferred revenue | 3,503 | ||
Cash and cash equivalents at beginning of period | 0 | ||
Cash and cash equivalents at end of period | $ 0 | ||
Effect of Change [Member] | ASU 2014-09 [Member] | Preferred Stock [Member] | |||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||
Net loss per share, basic and diluted (in dollars per share) | $ (0.12) | ||
Effect of Change [Member] | ASU 2014-09 [Member] | Research and Development Under Collaborative Agreement [Member] | |||
Condensed Consolidated Statements of Operations and Comprehensive Loss [Abstract] | |||
Revenue | $ (3,503) |
Investments and Fair Value Me27
Investments and Fair Value Measurements, Summary of Investments (Details) - USD ($) $ in Thousands | Mar. 31, 2018 | Dec. 31, 2017 |
Summary of Investments [Abstract] | ||
Cost | $ 175,410 | $ 202,100 |
Gross unrealized gains | 0 | 0 |
Gross unrealized losses | (382) | (337) |
Estimated fair value | 175,028 | 201,763 |
Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 169,962 | 170,569 |
Gross unrealized gains | 0 | 0 |
Gross unrealized losses | (355) | (265) |
Estimated fair value | 169,607 | 170,304 |
Securities with Maturity of One to Two Years [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 5,448 | 31,531 |
Gross unrealized gains | 0 | 0 |
Gross unrealized losses | (27) | (72) |
Estimated fair value | 5,421 | 31,459 |
Corporate Debt Securities [Member] | Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 101,180 | 132,434 |
Gross unrealized gains | 0 | 0 |
Gross unrealized losses | (220) | (206) |
Estimated fair value | 100,960 | 132,228 |
Corporate Debt Securities [Member] | Securities with Maturity of One to Two Years [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 2,919 | 8,267 |
Gross unrealized gains | 0 | 0 |
Gross unrealized losses | (23) | (35) |
Estimated fair value | 2,896 | 8,232 |
Debt Securities Issued by U.S. Government Agencies [Member] | Securities with Maturity of One Year or Less [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 68,782 | 38,135 |
Gross unrealized gains | 0 | 0 |
Gross unrealized losses | (135) | (59) |
Estimated fair value | 68,647 | 38,076 |
Debt Securities Issued by U.S. Government Agencies [Member] | Securities with Maturity of One to Two Years [Member] | ||
Summary of Investments [Abstract] | ||
Cost | 2,529 | 23,264 |
Gross unrealized gains | 0 | 0 |
Gross unrealized losses | (4) | (37) |
Estimated fair value | $ 2,525 | $ 23,227 |
Investments and Fair Value Me28
Investments and Fair Value Measurements, Fair Value Measurements (Details) - USD ($) | Mar. 31, 2018 | Dec. 31, 2017 | |
Fair Value Measurements [Abstract] | |||
Transfers from Level 1 to Level 2 | $ 0 | $ 0 | |
Transfers from Level 2 to Level 1 | 0 | 0 | |
Recurring Basis [Member] | |||
Fair Value Measurements [Abstract] | |||
Cash equivalents | [1] | 65,963,000 | 48,430,000 |
Total | 240,991,000 | 250,193,000 | |
Recurring Basis [Member] | Corporate Debt Securities [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | [2] | 103,856,000 | 140,460,000 |
Recurring Basis [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | [2] | 71,172,000 | 61,303,000 |
Recurring Basis [Member] | Quoted Prices in Active Markets (Level 1) [Member] | |||
Fair Value Measurements [Abstract] | |||
Cash equivalents | 65,963,000 | 48,430,000 | |
Total | 65,963,000 | 48,430,000 | |
Recurring Basis [Member] | Quoted Prices in Active Markets (Level 1) [Member] | Corporate Debt Securities [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | 0 | 0 | |
Recurring Basis [Member] | Quoted Prices in Active Markets (Level 1) [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | 0 | 0 | |
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | |||
Fair Value Measurements [Abstract] | |||
Cash equivalents | 0 | 0 | |
Total | 175,028,000 | 201,763,000 | |
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | Corporate Debt Securities [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | 103,856,000 | 140,460,000 | |
Recurring Basis [Member] | Significant Other Observable Inputs (Level 2) [Member] | Debt Securities Issued by U.S. Government Agencies [Member] | |||
Fair Value Measurements [Abstract] | |||
Available-for-sale securities | 71,172,000 | 61,303,000 | |
Recurring Basis [Member] | Significant Unobservable Inputs (Level 3) [Member] | |||
Fair Value Measurements [Abstract] | |||
Total | $ 0 | $ 0 | |
[1] | Included in cash and cash equivalents on our condensed consolidated balance sheets. | ||
[2] | Included in short-term investments on our condensed consolidated balance sheets. |
Strategic Collaboration with 29
Strategic Collaboration with Novartis (Details) $ in Thousands | Jul. 19, 2017USD ($) | Jul. 31, 2017USD ($) | Jan. 31, 2017DrugPerformanceObligation | Mar. 31, 2018USD ($) | Mar. 31, 2017USD ($) | Dec. 31, 2017USD ($) |
Strategic Collaboration with Novartis [Abstract] | ||||||
Proceeds from sale of common stock to Novartis in a private placement | $ 50,000 | |||||
Revenue earned | $ 17,108 | $ 6,094 | ||||
Revenue recognized from deferred revenue | 17,100 | |||||
AKCEA-APO(a)-L [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Maximum amount of payments receivable for milestones | 600,000 | |||||
Maximum amount of payments receivable for development milestones | 25,000 | |||||
Maximum amount of payments receivable for regulatory milestones | 290,000 | |||||
Maximum amount of payments receivable for commercialization milestones | $ 285,000 | |||||
Royalty percentage received on sales of drug | 20.00% | |||||
Development Services for AKCEA-APO(a)-L [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Transaction price | 64,000 | |||||
Delivery of AKCEA-APO(a)-L [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Transaction price | 1,500 | |||||
AKCEA-APOCIII-L [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Maximum amount of payments receivable for milestones | $ 530,000 | |||||
Maximum amount of payments receivable for development milestones | 25,000 | |||||
Maximum amount of payments receivable for regulatory milestones | 240,000 | |||||
Maximum amount of payments receivable for commercialization milestones | $ 265,000 | |||||
Royalty percentage received on sales of drug | 20.00% | |||||
Development Services for AKCEA-APOCIII-L [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Transaction price | 40,100 | |||||
Delivery of AKCEA-APOCIII-L [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Transaction price | 2,800 | |||||
R&D Under Collaborative Agreement [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Revenue earned | $ 17,108 | 6,094 | ||||
Novartis [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Upfront payment received | 75,000 | |||||
Portion of upfront payment retained | 60,000 | |||||
Portion of upfront payment paid as sublicense fee to Ionis | 15,000 | |||||
License fee receivable per drug | 150,000 | |||||
Next prospective milestone | $ 25,000 | |||||
Percentage of license fees, milestone payments and royalties paid as sublicense fee to Ionis | 50.00% | |||||
Number of separate performance obligations | PerformanceObligation | 4 | |||||
Transaction price | 108,400 | |||||
Premium received on shares issued by Ionis | 28,400 | |||||
Potential premium received if Ionis common stock is purchased in the future | 5,000 | |||||
Number of months from inception of agreement for IPO to be completed | 15 months | |||||
Remaining performance obligation | $ 56,700 | |||||
Proceeds from sale of common stock to Novartis in a private placement | $ 50,000 | |||||
Concentration percentage | 100.00% | |||||
Deferred revenue | $ 56,700 | $ 70,700 | ||||
Novartis [Member] | Minimum [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Number of drugs with exclusive option that could be exercised | Drug | 1 | |||||
Novartis [Member] | AKCEA-APO(a)-L [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Period of performance | 2 years | |||||
Novartis [Member] | AKCEA-APOCIII-L [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Period of performance | 2 years 6 months | |||||
Novartis [Member] | R&D Under Collaborative Agreement [Member] | ||||||
Strategic Collaboration with Novartis [Abstract] | ||||||
Revenue earned | $ 17,100 | $ 6,100 |
Development, Commercializatio30
Development, Commercialization and License Agreement and Services Agreement with Ionis (Details) $ in Thousands | 3 Months Ended | |
Mar. 31, 2018USD ($)PatientPayment | Mar. 31, 2017USD ($) | |
Operating Expenses Generated by Transactions with Ionis [Abstract] | ||
Payable balance to Ionis at beginning of the period | $ 14,365 | |
Total amount payable to Ionis at period end | $ 27,737 | |
Ionis [Member] | Development, Commercialization and License Agreement [Member] | ||
Development, Commercialization and License Agreement with Ionis [Abstract] | ||
Royalty percentage paid on sales of lipid drug | 20.00% | |
First annual sales threshold | $ 500,000 | |
Second annual sales threshold | 1,000,000 | |
Third annual sales threshold | 2,000,000 | |
Sales milestone payment | $ 50,000 | |
Number of quarters in which equal payments are made | Payment | 12 | |
Ionis [Member] | Development, Commercialization and License Agreement [Member] | Lipid Drug for Rare Disease Indication [Member] | Maximum [Member] | ||
Development, Commercialization and License Agreement with Ionis [Abstract] | ||
Number of patients worldwide | Patient | 500,000 | |
Number of patients for Phase 3 program | Patient | 1,000 | |
Number of years of treatment | 2 years | |
Ionis [Member] | Development, Commercialization and License Agreement [Member] | Lipid Drug for a Broad Disease Patient Population [Member] | Minimum [Member] | ||
Development, Commercialization and License Agreement with Ionis [Abstract] | ||
Number of patients worldwide | Patient | 500,000 | |
Number of patients for Phase 3 program | Patient | 1,000 | |
Number of years of treatment | 2 years | |
Ionis [Member] | Services Agreement [Member] | ||
Services Agreement with Ionis [Abstract] | ||
Payment term after receipt of invoice | 30 days | |
Term of extension for services agreement | 6 months | |
Operating Expenses Generated by Transactions with Ionis [Abstract] | ||
Services performed by Ionis | $ 1,945 | $ 2,957 |
Active pharmaceutical ingredient manufactured by Ionis | 5,229 | 3,083 |
Sublicensing expenses | 0 | 48,394 |
Out-of-pocket expenses paid by Ionis | 6,236 | 8,868 |
Total expenses generated by transactions with Ionis | 13,410 | 63,302 |
Payable balance to Ionis at beginning of the period | 14,365 | 24,355 |
Prepaid amounts to Ionis | 0 | 3,005 |
Less: total amounts paid to Ionis during the period | (38) | (42,268) |
Less: non-cash sublicensing expenses | 0 | (33,394) |
Total amount payable to Ionis at period end | $ 27,737 | $ 15,000 |
Stock-Based Compensation, Stock
Stock-Based Compensation, Stock Plans (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | |
Dec. 31, 2017 | May 31, 2017 | Mar. 31, 2018 | |
Stock Plans [Abstract] | |||
Accrued liability for contributions to employee stock purchase plan | $ 144 | ||
2015 Equity Incentive Plan [Member] | |||
Stock Plans [Abstract] | |||
Number of shares of common stock reserved for issuance (in shares) | 13,500,000 | 8,500,000 | |
Number of shares authorized for issuance pursuant to stock awards (in shares) | 8,500,000 | ||
Number of additional shares authorized for issuance pursuant to stock awards (in shares) | 5,000,000 | ||
2015 Equity Incentive Plan [Member] | Stock Options [Member] | |||
Stock Plans [Abstract] | |||
Options outstanding (in shares) | 9,298,024 | ||
Options exercisable (in shares) | 3,276,937 | ||
Number of shares available for future grant (in shares) | 4,169,447 | ||
2015 Equity Incentive Plan [Member] | RSUs [Member] | |||
Stock Plans [Abstract] | |||
Restricted stock units outstanding (in shares) | 32,529 | ||
2017 Employee Stock Purchase Plan [Member] | |||
Stock Plans [Abstract] | |||
Number of shares of common stock reserved for issuance (in shares) | 1,165,416 | ||
Percentage of shares outstanding used to calculate annual increase in number of shares that can be issued under ESPP | 1.00% | ||
Annual maximum increase in number of shares that can be issued under ESPP (in shares) | 500,000 | ||
Shares issued under ESPP (in shares) | 15,732 | ||
Number of shares that may be issued (in shares) | 1,149,684 |
Stock-Based Compensation, Sto32
Stock-Based Compensation, Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2018 | Mar. 31, 2017 | |
Stock-Based Compensation Expense [Abstract] | ||
Stock-based compensation expense | $ 6,384 | $ 3,180 |
Research and Development Expenses [Member] | ||
Stock-Based Compensation Expense [Abstract] | ||
Stock-based compensation expense | 2,314 | 1,600 |
General and Administrative Expenses [Member] | ||
Stock-Based Compensation Expense [Abstract] | ||
Stock-based compensation expense | $ 4,070 | $ 1,580 |
Accumulated Other Comprehensi33
Accumulated Other Comprehensive Loss (Details) $ in Thousands | 3 Months Ended | |
Mar. 31, 2018USD ($) | ||
Accumulated Other Comprehensive Loss [Roll Forward] | ||
Beginning balance | $ 167,825 | |
Net other comprehensive income (loss) | (17) | |
Ending balance | 146,300 | |
Tax benefit included in other comprehensive income (loss) | 0 | |
Accumulated Other Comprehensive Loss [Member] | ||
Accumulated Other Comprehensive Loss [Roll Forward] | ||
Beginning balance | (451) | |
Ending balance | (468) | |
Unrealized Gains (Losses) on Securities [Member] | ||
Accumulated Other Comprehensive Loss [Roll Forward] | ||
Other comprehensive loss before reclassifications, net of tax | (45) | [1] |
Currency Translation Adjustment [Member] | ||
Accumulated Other Comprehensive Loss [Roll Forward] | ||
Other comprehensive loss before reclassifications, net of tax | $ 28 | |
[1] | There was no tax benefit for other comprehensive income (loss) for the three months ended March 31, 2018. |
Basic and Diluted Net Loss Pe34
Basic and Diluted Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | |
Mar. 31, 2018 | Mar. 31, 2017 | |
Basic and Diluted Net Loss Per Share [Abstract] | ||
Net loss | $ (29,627) | $ (63,856) |
Preferred Stock [Member] | ||
Basic and Diluted Net Loss Per Share [Abstract] | ||
Net loss | $ 0 | $ (63,856) |
Weighted-average shares outstanding (in shares) | 0 | 28,884,540 |
Basic loss per share (in dollars per share) | $ 0 | $ (2.21) |
Common Stock [Member] | ||
Basic and Diluted Net Loss Per Share [Abstract] | ||
Net loss | $ (29,627) | $ 0 |
Weighted-average shares outstanding (in shares) | 66,616,337 | 0 |
Basic loss per share (in dollars per share) | $ (0.44) | $ 0 |
Initial Public Offering (Detail
Initial Public Offering (Details) $ in Millions | Jul. 19, 2017USD ($)shares |
Initial Public Offering [Abstract] | |
Net proceeds from sale of common stock in IPO | $ 182.3 |
Gross proceeds from sale of common stock in IPO | 132.3 |
Investment by Ionis | 25 |
Proceeds from sale of common stock to Novartis in a private placement | $ 50 |
Shares issued upon conversion of Series A Convertible Preferred Stock (in shares) | shares | 28,884,540 |
Principal and accrued interest from line of credit converted | $ 106 |
Shares issued upon conversion of line of credit (in shares) | shares | 13,438,339 |
IPO [Member] | |
Initial Public Offering [Abstract] | |
Shares issued (in shares) | shares | 17,968,750 |
Private Placement [Member] | |
Initial Public Offering [Abstract] | |
Shares issued (in shares) | shares | 6,250,000 |
Subsequent Events (Details)
Subsequent Events (Details) $ in Millions | Apr. 17, 2018USD ($)Milestoneshares | Apr. 05, 2018USD ($)ft²Option | Apr. 30, 2018USD ($) | Apr. 16, 2018shares | Mar. 31, 2018shares | Dec. 31, 2017shares |
Development, Commercialization, Collaboration and License Agreement [Abstract] | ||||||
Ownership percentage by Ionis | 68.00% | |||||
Common stock, shares authorized (in shares) | shares | 100,000,000 | 100,000,000 | ||||
Subsequent Event [Member] | ||||||
Development, Commercialization, Collaboration and License Agreement [Abstract] | ||||||
Upfront licensing fee paid to Ionis | $ 150 | |||||
Shares issued in consideration of upfront licensing fee | shares | 8,000,000 | |||||
Number of sales milestones | Milestone | 7 | |||||
Annual worldwide net sales required for subsequent milestone payments to be paid in cash | $ 750 | |||||
Percentage of profits and losses from development and commercialization of inotersen paid to Ionis, tier 1 | 60.00% | |||||
Percentage of profits and losses from development and commercialization of inotersen retained, tier 1 | 40.00% | |||||
Percentage of profits and losses from development and commercialization of inotersen paid to Ionis, tier 2 | 50.00% | |||||
Percentage of profits and losses from development and commercialization of inotersen retained, tier 2 | 50.00% | |||||
Percentage of profits and losses from development and commercialization of AKCEA-TTR-L paid to Ionis | 50.00% | |||||
Percentage of profits and losses from development and commercialization of AKCEA-TTR-L retained | 50.00% | |||||
Notice period for termination of agreement | 90 days | |||||
Shares purchased by Ionis (in shares) | shares | 10,700,000 | |||||
Stock purchased by Ionis | $ 200 | $ 200 | ||||
Ownership percentage by Ionis | 75.00% | |||||
Common stock, shares authorized (in shares) | shares | 125,000,000 | 100,000,000 | ||||
Subsequent Event [Member] | Inotersen [Member] | ||||||
Development, Commercialization, Collaboration and License Agreement [Abstract] | ||||||
Maximum amount of payments payable for milestones | $ 110 | |||||
Subsequent Event [Member] | AKCEA-TTR-L [Member] | ||||||
Development, Commercialization, Collaboration and License Agreement [Abstract] | ||||||
Maximum amount of payments payable for milestones | 145 | |||||
Subsequent Event [Member] | Inotersen and AKCEA-TTR-L [Member] | ||||||
Development, Commercialization, Collaboration and License Agreement [Abstract] | ||||||
Maximum amount of payments payable for milestones | $ 1,300 | |||||
Subsequent Event [Member] | Office Space for Corporate Headquarters [Member] | ||||||
Leases, Operating [Abstract] | ||||||
Area of office space under lease agreement | ft² | 30,175 | |||||
Initial term of lease | 123 months | |||||
Number of renewal options | Option | 1 | |||||
Term of renewal period | 5 years | |||||
Annual lease payments due in 2018 | $ 0.8 | |||||
Annual lease payments due in 2019 | 2.2 | |||||
Annual lease payments due in 2020 | 2.3 | |||||
Annual lease payments due in 2021 | 2.3 | |||||
Annual lease payments due in 2022 | 2.3 | |||||
Annual lease payments due thereafter | $ 14.2 | |||||
Period of free rent | 3 months | |||||
Tenant improvement allowance | $ 3.8 | |||||
Initial letter of credit to secure lease obligation | 2.4 | |||||
Letter of credit to secure lease obligation on third anniversary of rent commencement date | 1.8 | |||||
Letter of credit to secure lease obligation on fifth anniversary of rent commencement date | $ 1.2 |